Spinal Muscular Atrophy

The FDA has approved Evrysdi in tablet form for patients with spinal muscular atrophy aged 2 years and older who weigh more than 44 pounds, according to the manufacturer.

A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully human monoclonal antibody to treat individuals with spinal muscular atrophy.

Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with spinal muscular atrophy type 2 increased mobility and slowed disease progression, according to a press release from Novartis.

Disease-modifying therapies introduced in Italy led to better outcomes and survival rates in children born after 2016 with type 1 spinal muscular atrophy compared with those born prior to then, new data published in EClinicalMedicine show.

Topline results from the phase 3 SAPPHIRE clinical trial showed that an investigational muscle-targeted therapy improved motor function in patients with spinal muscular atrophy compared with placebo as early as week 8.

Topline data from an ongoing clinical trial of a higher dose regimen of Spinraza in treatment-naive, symptomatic infants with spinal muscular atrophy showed significantly improved motor function at 6 months.

A Georgia-based biotechnology company has secured a U.S. patent for an exosome-based therapy to treat neuroinflammation that occurs in a range of neurodegenerative disease, according to the manufacturer.

An investigational, fully human monoclonal antibody developed as a muscle-targeted therapy for spinal muscular atrophy showed sustained motor function improvements at 48 months, according to the manufacturer.

Newly released data from the open-label extension of the FIREFISH study confirmed sustained safety and efficacy of Evrysdi in children with type 1 spinal muscular atrophy over 5 years.